ES3050313T3 - Liver-specific tropism of adeno-associated viruses - Google Patents
Liver-specific tropism of adeno-associated virusesInfo
- Publication number
- ES3050313T3 ES3050313T3 ES19799988T ES19799988T ES3050313T3 ES 3050313 T3 ES3050313 T3 ES 3050313T3 ES 19799988 T ES19799988 T ES 19799988T ES 19799988 T ES19799988 T ES 19799988T ES 3050313 T3 ES3050313 T3 ES 3050313T3
- Authority
- ES
- Spain
- Prior art keywords
- seq
- amino acid
- aav
- capsid protein
- hepatic
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Active
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14145—Special targeting system for viral vectors
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Medicinal Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Crystallography & Structural Chemistry (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862670543P | 2018-05-11 | 2018-05-11 | |
| US201962841179P | 2019-04-30 | 2019-04-30 | |
| PCT/US2019/031851 WO2019217911A1 (fr) | 2018-05-11 | 2019-05-10 | Tropisme hépato-spécifique de virus adéno-associés |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ES3050313T3 true ES3050313T3 (en) | 2025-12-19 |
Family
ID=68468421
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ES19799988T Active ES3050313T3 (en) | 2018-05-11 | 2019-05-10 | Liver-specific tropism of adeno-associated viruses |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US11987801B2 (fr) |
| EP (1) | EP3790567B1 (fr) |
| JP (2) | JP7438981B2 (fr) |
| KR (1) | KR102946842B1 (fr) |
| CN (1) | CN112368391A (fr) |
| AU (1) | AU2019264991B2 (fr) |
| BR (1) | BR112020022858A2 (fr) |
| CA (1) | CA3100006A1 (fr) |
| DK (1) | DK3790567T3 (fr) |
| ES (1) | ES3050313T3 (fr) |
| IL (1) | IL278598B2 (fr) |
| SA (1) | SA520420518B1 (fr) |
| WO (1) | WO2019217911A1 (fr) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3100006A1 (fr) | 2018-05-11 | 2019-11-14 | Massachusetts Eye And Ear Infirmary | Tropisme hepato-specifique de virus adeno-associes |
| EP3715358A1 (fr) * | 2019-03-28 | 2020-09-30 | Universität zu Köln | Proteines de capside de virus adeno-associées mutantes, particule d'aav les contenant et thérapie génique par vecteurs aav administrée par le foie |
| CA3153972A1 (fr) | 2019-09-09 | 2021-03-18 | Massachusetts Eye And Ear Infirmary | Methodes et compositions de modulation de l'interaction entre un virus adeno-associe (aav) et le recepteur d'aav (aavr) a des fins de bio-distribution modifiee d'aav |
| AU2020368539A1 (en) | 2019-10-16 | 2022-04-28 | Massachusetts Institute Of Technology | Engineered muscle targeting compositions |
| WO2021222831A2 (fr) * | 2020-05-01 | 2021-11-04 | The Broad Institute, Inc. | Compositions de système nerveux central génétiquement modifiées |
| CN111876432B (zh) * | 2020-07-29 | 2022-10-28 | 舒泰神（北京）生物制药股份有限公司 | 一组肝靶向新型腺相关病毒的获得及其应用 |
| AU2021378075B2 (en) | 2020-11-11 | 2025-03-27 | Borea Therapeutics S.R.L. | Modified viral particles for gene therapy |
| WO2022150634A2 (fr) | 2021-01-08 | 2022-07-14 | Massachusetts Eye And Ear Infirmary | Méthodes et compositions pour moduler l'interaction entre un virus adéno-associé (vaa) et le récepteur de vaa (aavr) à des fins de bio-distribution modifiée de vaa |
| US20240115734A1 (en) * | 2021-02-09 | 2024-04-11 | Affinia Therapeutics Inc. | Recombinant aavs with improved tropism and specificity |
| US20240352077A1 (en) * | 2021-08-25 | 2024-10-24 | Children's Medical Research Institute | Modified aav capsids and vectors |
| WO2023039480A2 (fr) * | 2021-09-08 | 2023-03-16 | The Broad Institute, Inc. | Compositions modifiées pour le ciblage du système nerveux central |
| KR20240095539A (ko) | 2021-10-08 | 2024-06-25 | 디노 테라퓨틱스, 인코포레이티드 | 캡시드 변이체 및 이의 사용 방법 |
| WO2024238807A2 (fr) | 2023-05-16 | 2024-11-21 | Affinia Therapeutics Inc. | Aav de recombinaison à tropisme et spécificité améliorés |
| WO2025059678A1 (fr) * | 2023-09-15 | 2025-03-20 | Ohio State Innovation Foundation | Nouveaux sérotypes de capside ingénierisés de vecteurs viraux adéno-associés recombinés à tropisme hépatique réduit |
| WO2025137378A1 (fr) * | 2023-12-20 | 2025-06-26 | Aavantibio, Inc. | Ingénierie de capside d'aav pour thérapie génique cardiaque et/ou musculo-squelettique |
| US20250276095A1 (en) | 2024-03-04 | 2025-09-04 | Kate Therapeutics, Inc. | Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy |
| WO2025194042A1 (fr) | 2024-03-14 | 2025-09-18 | Affinia Therapeutics Inc. | Système plasmidique pour la production d'un virus adénoassocié recombinant |
| WO2025226842A1 (fr) | 2024-04-24 | 2025-10-30 | Kate Therapeutics, Inc. | Contrôle d'expression par des arnmi exprimés par drg |
| WO2025226841A1 (fr) | 2024-04-24 | 2025-10-30 | Kate Therapeutics, Inc. | Approche de thérapie génique pour le traitement de troubles associés à tnnt2 |
| WO2025227063A1 (fr) | 2024-04-26 | 2025-10-30 | Affinia Therapeutics Inc. | Vaa recombinants à tropisme et spécificité améliorés |
| WO2025235491A1 (fr) | 2024-05-07 | 2025-11-13 | Affinia Therapeutics Inc. | Aav recombinant pour le traitement de maladies cardiaques |
| WO2026011008A1 (fr) | 2024-07-02 | 2026-01-08 | Kate Therapeutics, Inc. | Contrôle d'expression par miarn exprimés par les muscles squelettiques |
| WO2026011009A1 (fr) | 2024-07-02 | 2026-01-08 | Kate Therapeutics, Inc. | Compositions et procédés pour troubles musculaires |
| WO2026030242A1 (fr) | 2024-07-29 | 2026-02-05 | Kate Therapeutics, Inc. | Compositions de virus adéno-associés pour le traitement de la dystrophie musculaire facio-scapulo-humérale |
| WO2026030244A1 (fr) | 2024-07-30 | 2026-02-05 | Kate Therapeutics, Inc. | Compositions de virus adéno-associés pour le traitement de la dystrophie musculaire des ceintures de type 2a |
| WO2026050402A1 (fr) | 2024-08-29 | 2026-03-05 | Kate Therapeutics, Inc. | Capsides tropiques musculaires dé-ciblées du foie |
Family Cites Families (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2345731B1 (fr) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Variantes des virus associés aux adénovirus (AAV), séquences, vecteurs les contenant et leur utilisation |
| JP5136766B2 (ja) * | 2004-12-15 | 2013-02-06 | ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | キメラベクター |
| WO2010093784A2 (fr) * | 2009-02-11 | 2010-08-19 | The University Of North Carolina At Chapel Hill | Vecteurs viraux modifiés et procédés de fabrication et d'utilisation de ceux-ci |
| CN105755044A (zh) * | 2011-04-22 | 2016-07-13 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体及其使用方法 |
| JP5915467B2 (ja) | 2012-08-30 | 2016-05-11 | 株式会社島津製作所 | 液体クロマトグラフ検出器用送液管及び液体クロマトグラフ |
| ES2926774T3 (es) | 2013-03-15 | 2022-10-28 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores AAV de doble unión de glicano |
| EP3003391B1 (fr) * | 2013-05-31 | 2021-09-22 | The Regents of The University of California | Variants de virus adéno-associés et leurs méthodes d'utilisation |
| WO2015048534A1 (fr) | 2013-09-26 | 2015-04-02 | University Of Florida Research Foundation, Inc. | Bibliothèque de capsides aav combinatoire synthétique pour la thérapie génique ciblée |
| CN106232618A (zh) | 2013-10-11 | 2016-12-14 | 马萨诸塞眼科耳科诊所 | 预测祖先病毒序列的方法及其用途 |
| US9474181B2 (en) | 2013-12-23 | 2016-10-18 | Dell Products, L.P. | Combined power and management module for use in a rack-configured information handling system |
| WO2015164757A1 (fr) * | 2014-04-25 | 2015-10-29 | Oregon Health & Science University | Procedes de cartographie d'epitopes d'anticorps de neutralisation virale |
| JP6475998B2 (ja) | 2015-02-10 | 2019-02-27 | デクセリアルズ株式会社 | タッチパッド用アンテナ装置、及び電子機器 |
| CN108603235A (zh) | 2015-07-30 | 2018-09-28 | 马萨诸塞眼科耳科诊所 | 祖先病毒序列及其用途 |
| EP3384034B1 (fr) | 2015-12-02 | 2020-07-08 | The Board of Trustees of the Leland Stanford Junior University | Nouveaux capsides de virus adéno-associés recombinants avec du tropisme pour les muscles sqeletiques humains amélioré |
| SG10201913266UA (en) * | 2015-12-11 | 2020-02-27 | Massachusetts Eye & Ear Infirmary | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
| JP6947739B2 (ja) | 2016-02-16 | 2021-10-13 | ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー | 既存のヒト中和抗体に対して耐性である新規組換えアデノ随伴ウイルスカプシド |
| JP6936788B2 (ja) | 2016-04-01 | 2021-09-22 | 株式会社カネカ | 軽量化及びリブ設計が可能な樹脂組成物による表面性が良好な射出発泡成形体 |
| IL305149A (en) * | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| AU2017341849B2 (en) | 2016-10-13 | 2024-03-21 | University Of Massachusetts | AAV capsid designs |
| US11905312B2 (en) | 2017-02-15 | 2024-02-20 | The University Of North Carolina At Chapel Hill | Methods and compositions for gene transfer across the vasculature |
| CA3061968A1 (fr) | 2017-05-10 | 2018-11-15 | Massachusetts Eye And Ear Infirmary | Procedes et compositions pour modifier la dependance a une proteine activant l'assemblage (aap) de virus |
| US11247659B2 (en) | 2017-08-08 | 2022-02-15 | Aisin Seiki Kabushiki Kaisha | Vehicle control apparatus |
| CN111770999A (zh) | 2017-11-27 | 2020-10-13 | 4D分子治疗有限公司 | 腺相关病毒变体衣壳和用于抑制血管生成的应用 |
| CA3100006A1 (fr) | 2018-05-11 | 2019-11-14 | Massachusetts Eye And Ear Infirmary | Tropisme hepato-specifique de virus adeno-associes |
| WO2020041498A1 (fr) | 2018-08-21 | 2020-02-27 | Massachusetts Eye And Ear Infirmary | Compositions et procédés pour moduler l'efficacité de transduction de virus adéno-associés |
| CA3153972A1 (fr) | 2019-09-09 | 2021-03-18 | Massachusetts Eye And Ear Infirmary | Methodes et compositions de modulation de l'interaction entre un virus adeno-associe (aav) et le recepteur d'aav (aavr) a des fins de bio-distribution modifiee d'aav |
| WO2022150634A2 (fr) | 2021-01-08 | 2022-07-14 | Massachusetts Eye And Ear Infirmary | Méthodes et compositions pour moduler l'interaction entre un virus adéno-associé (vaa) et le récepteur de vaa (aavr) à des fins de bio-distribution modifiée de vaa |
-
2019
- 2019-05-10 CA CA3100006A patent/CA3100006A1/fr active Pending
- 2019-05-10 IL IL278598A patent/IL278598B2/en unknown
- 2019-05-10 DK DK19799988.1T patent/DK3790567T3/da active
- 2019-05-10 CN CN201980042499.3A patent/CN112368391A/zh active Pending
- 2019-05-10 US US17/053,412 patent/US11987801B2/en active Active
- 2019-05-10 ES ES19799988T patent/ES3050313T3/es active Active
- 2019-05-10 AU AU2019264991A patent/AU2019264991B2/en active Active
- 2019-05-10 BR BR112020022858-3A patent/BR112020022858A2/pt unknown
- 2019-05-10 JP JP2020563602A patent/JP7438981B2/ja active Active
- 2019-05-10 KR KR1020207035395A patent/KR102946842B1/ko active Active
- 2019-05-10 WO PCT/US2019/031851 patent/WO2019217911A1/fr not_active Ceased
- 2019-05-10 EP EP19799988.1A patent/EP3790567B1/fr active Active
-
2020
- 2020-11-10 SA SA520420518A patent/SA520420518B1/ar unknown
-
2024
- 2024-02-14 JP JP2024020029A patent/JP7727029B2/ja active Active
- 2024-04-12 US US18/633,726 patent/US20240344085A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| DK3790567T3 (da) | 2025-11-03 |
| KR102946842B1 (ko) | 2026-04-01 |
| JP2021522827A (ja) | 2021-09-02 |
| BR112020022858A2 (pt) | 2021-02-23 |
| EP3790567A4 (fr) | 2021-12-01 |
| AU2019264991B2 (en) | 2025-12-04 |
| EP3790567B1 (fr) | 2025-08-06 |
| KR20210010491A (ko) | 2021-01-27 |
| CA3100006A1 (fr) | 2019-11-14 |
| SA520420518B1 (ar) | 2024-03-17 |
| IL278598B2 (en) | 2025-11-01 |
| EP3790567A1 (fr) | 2021-03-17 |
| JP7727029B2 (ja) | 2025-08-20 |
| WO2019217911A1 (fr) | 2019-11-14 |
| US11987801B2 (en) | 2024-05-21 |
| AU2019264991A1 (en) | 2020-11-26 |
| IL278598B1 (en) | 2025-07-01 |
| JP2024073426A (ja) | 2024-05-29 |
| JP7438981B2 (ja) | 2024-02-27 |
| IL278598A (fr) | 2021-03-01 |
| US20230051611A1 (en) | 2023-02-16 |
| US20240344085A1 (en) | 2024-10-17 |
| CN112368391A (zh) | 2021-02-12 |
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